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A group of Alzheimer’s experts and health advocates called on the F.D.A. to withdraw its approval of the drug, the latest of several setbacks for the treatment.
Biogen slashed the price of its controversial new Alzheimer’s drug Aduhelm on Monday as the drug faces weak sales and mounting criticism.
The price was reduced to $28,200 a year from $56,000 on the same day that a group of Alzheimer’s experts and health advocates called on the Food and Drug Administration to pull the drug off the market and said they were supporting an effort to file a formal petition with the agency to withdraw it.
“The F.D.A.’s decision to approve Aduhelm is indefensible in both scientific and clinical terms,” said a statement signed by 18 scientists, most of them doctors. “This drug should be withdrawn from the market immediately.”
The agency approved Aduhelm in June, even though a council of senior F.D.A. officials, an advisory committee of outside experts and many Alzheimer’s specialists said the scientific evidence showed that the drug did not provide a clear benefit to patients and that it carried risks of dangerous side effects.
Major health systems, including Cleveland Clinic, Mount Sinai Health System, Mass General Brigham and the Department of Veterans Affairs have declined to offer Aduhelm, citing questions about its benefits and risks. In October, Biogen reported that Aduhelm had brought in just $1.9 million in revenue through September, a strikingly small amount given that about 1.5 million Americans have the mild Alzheimer’s-related dementia that makes them eligible for the drug.
In a statement about the price-cutting on Monday, Michel Vounatsos, Biogen’s chief executive officer, said the company had “listened to the feedback of our stakeholders” and believed that “too many patients are not being offered the choice of Aduhelm due to financial considerations.”
But Brian Skorney, an analyst at Robert W. Baird & Company, said after the price announcement: “For the broad majority of people who are critical of Aduhelm, $56,000 and $28,000 are both exceptionally high prices for a drug that a lot of people perceive doesn’t work at all.”
The statement from Alzheimer’s experts and health advocates calling on the F.D.A. to withdraw Aduhelm grew out of a three-hour video meeting among the scientists last week.
“We’re not just saying the approval was probably the worst decision the F.D.A. ever made,” Dr. Peter Whitehouse, a neurologist and Alzheimer’s expert at Case Western Reserve University who led the Dec. 15 meeting, told attendees during the session. “It’s so bad that we should advocate for withdrawal.”
The doctors and scientists who signed the statement also agreed to provide their expertise to support the filing of a citizen petition, a formal process to seek reversal of the F.D.A.’s decision. The petition will be filed by the Right Care Alliance, a coalition of clinicians, patients and community members, which is also circulating a pledge for physicians who promise not to prescribe Aduhelm and for patients and family members who say they will not request it.
Dr. Vikas Saini, chairman of the Right Care Alliance and president of the Lown Institute, a health care think tank, said that while the citizen petition process can take months or years, it can prompt F.D.A. action.
Dr. Saini, who helped organize last week’s meeting, said that out of 45 citizen petitions filed since 1971 by the group Public Citizen, in 27 cases the F.D.A. withdrew drugs from the market and in seven other cases the agency sharply restricted a drug’s use.
In response to the call to withdraw approval of Aduhelm, also known by its scientific name, aducanumab, an F.D.A. spokeswoman said that “the data set for Aduhelm was very complex, and our review has been thorough.”
The spokeswoman also said that “careful analyses of the Phase 2 and Phase 3 clinical trials support the conclusion that it is likely that Aduhelm provides clinical benefit — although the data currently available do not provide substantial evidence of effectiveness on clinical benefit.”
Aduhelm has also been encountering resistance in other countries. On Friday, reviewers at the European Union’s drug regulator, the European Medicines Agency, recommended against approving Aduhelm, a decision that Biogen said it would ask to be re-examined.
Several months ago, leading Canadian Alzheimer’s research organizations said that approving the drug in Canada “cannot be justified.”
Dr. Howard Chertkow, scientific director of the Canadian Consortium on Neurodegeneration in Aging, was one of three Alzheimer’s experts from outside the United States who signed the statement calling on the F.D.A. to withdraw the drug. “We feel it’s an abrogation of our responsibility as physicians to allow a marginal medication with a very high cost associated with it to come into the country, so we’re quite active in trying to block what has happened in the states from occurring in Canada,” he said at last week’s meeting.
Two nearly identical clinical trials of Aduhelm, a monoclonal antibody given as a monthly infusion, were stopped early because an independent data monitoring committee concluded that the drug didn’t appear to be beneficial. A later analysis by Biogen found that participants receiving the high dose of the drug in one trial had experienced a very slight slowing of cognitive decline but that participants in the other trial had not benefited at all.
About 41 percent of patients receiving the high dose — the dose the F.D.A. approved — experienced brain swelling or brain bleeding, side effects that were often mild or asymptomatic, but were sometimes serious.
Monday’s statement also objects to the F.D.A.’s justification for its approval. Acknowledging there was insufficient evidence that Aduhelm would help patients, the agency greenlighted it under a program called “accelerated approval,” which allows authorization of drugs without proof of benefit for serious diseases that have few treatment options if the drug affects part of the disease’s biology (known as a surrogate endpoint) in a way that is “reasonably likely to predict clinical benefit.”
The F.D.A. based its approval on Aduhelm’s ability to reduce a protein called amyloid that forms plaques in the brains of people with Alzheimer’s. But experts say years of studies have not shown that reducing amyloid helps memory or thinking problems.
Approving the drug because of amyloid reduction “will have huge and wide-ranging negative implications for patients, families” and dementia research, Dr. Kenneth Langa, a professor of medicine at the University of Michigan, said in last week’s meeting.
The group’s statement, which includes a section for others to sign their names in support, plays on the F.D.A.’s language by calling for “accelerated withdrawal.”
“Reading the justification of the F.D.A. was like watching a ‘Saturday Night Live’ skit for data nerds.” Dr. Saini said. “I mean, I had trouble believing it.”
The agency’s approval requires that Biogen conduct another trial to see if Aduhelm works. In its response Monday, the F.D.A. spokeswoman said “we believe that the data support accelerated approval while holding the company accountable for conducting an additional study.” Last week, Biogen said that it had developed a protocol to get that trial completed in 2026, several years before the F.D.A.-imposed deadline.
Before he signed Monday’s statement, Dr. Sam Gandy, director of Mount Sinai’s Center for Cognitive Health, who helped organize the meeting, said that with advocacy groups like the Alzheimer’s Association still supporting the drug’s approval, he had a “concern that calling for something may trigger a backlash that undoes where we really want to go.”
But Dr. Jerry Avorn, a professor of medicine at Harvard Medical School who also signed the statement, said that while it was unlikely that calling for withdrawal would make F.D.A. officials say, “‘Yeah, they’re right, we goofed, we better undo this decision,’” the action was nonetheless important. “We’re taking a stand and saying the process was terrible and the outcome was terrible, and even if they don’t listen to it, it’ll be kind of a shot across the bow.”
Rebecca Robbins contributed reporting.